Avenue Therapeutics (ATXI) investor relations material

Executive summary

• Focused on developing therapies for neurologic diseases, with current candidates ATX-04 (for Pompe disease) and IV tramadol (for post-operative pain).

• In 2025, terminated the AJ201 license and sold Baergic Bio, narrowing the pipeline to two main candidates.

• Net loss for 2025 was $2.9 million, a significant improvement from $11.7 million in 2024.

• As of December 31, 2025, had $2.9 million in cash and cash equivalents and an accumulated deficit of $105.5 million.

• Substantial doubt exists about the ability to continue as a going concern without additional funding.

Financial highlights

• 2025 revenue was $1.4 million, primarily from payments related to the AJ201 license termination; no comparable revenue in 2024.

• Research and development expenses decreased to $1.0 million in 2025 from $6.6 million in 2024, mainly due to reduced AJ201 costs.

• General and administrative expenses were $3.7 million in 2025, down from $4.6 million in 2024.

• Net cash used in operating activities was $1.8 million in 2025, compared to $9.0 million in 2024.

• Net cash provided by financing activities was $2.1 million in 2025, mainly from ATM sales of common stock.

Outlook and guidance

• Plans to advance ATX-04 into late-stage clinical development for Pompe disease, pending FDA alignment and additional capital.

• Final agreement reached with FDA on Phase 3 safety study protocol for IV tramadol; study initiation depends on securing financing or partnership.

• Ongoing evaluation of opportunities to license or acquire additional product candidates.